The FDA has approved luspatercept-aamt (Reblozyl) for the treatment of anemia failing an erythropoiesis stimulating agent (ESA) and requiring 2 or more red blood cell (RBC) units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndromes with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T). Luspatercept is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.1
<.001 for both). In an analysis that applied the International Working Group (IWG) 2018 criteria, 19% of luspatercept-treated patients compared with 4% of patients on placebo had RBC-TI for ≥16 weeks during weeks 1 through 24; these rates were 28% and 7%, respectively, during weeks 1 through 48.
The study met an additional secondary endpoint, which was hematologic improvement-erythroid (HI-E) for ≥8 weeks, as assessed by IWG 2006 criteria. In weeks 1 through 24, HI-E responses occurred in 53% of patients on luspatercept compared with 12% of those in the placebo group. Fifty-nine percent of patients treated with luspatercept had an HI-E response in weeks 1 through 48 versus 17% of patients on placebo. During the double-blind period, no significant changes in neutrophil or platelet counts were observed.
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